Overview
Sponsor-declared trial summary
Neonatal hypoxic-ischaemic encephalopathy
The primary objective of this study is to test the safety and feasibility of a curative treatment with autologous cord blood stem cell in neonatal hypoxic-ischaemic encephalopathy.
Key facts
- Sponsor
- Centre Hospitalier Regional De Marseille
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Trial duration
- 5 Feb 2020 → ongoing
- Decision date (initial)
- 2024-09-10
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- Yes
- Funding sources
- DGOS_ PHRC
External identifiers
- EU CT number
- 2024-516421-30-00
- EudraCT number
- 2017-002957-12
- ClinicalTrials.gov
- NCT02881970
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Efficacy, Therapy, Safety
The primary objective of this study is to test the safety and feasibility of a curative treatment with autologous cord blood stem cell in neonatal hypoxic-ischaemic encephalopathy.
Secondary objectives 1
- The secondary objectives are to test the efficacy of this curative treatment with cell with neurogenic potential on the prevention of neurologic sequelae, as well as to test the optimum timing of cell preparation administration
Conditions and MedDRA coding
Neonatal hypoxic-ischaemic encephalopathy
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 6
- signs of encephalopathy within 6 hours of age (Sarnat and Sarnat classification, score ≥ 2)
- - ± abnormal electroencephalogram or aEEG within 6 hours of age
- - therapeutic hypothermia.
- - no maternal infection with VIH, HTLV 1 or 2, Hepatitis B or C virus
- maternal negative serology for syphilis
- written parental consent
Exclusion criteria 8
- major congenital anomalies, including severe metabolic diseases
- severe maternal-fetal infection responsible for anoxo-ischemia, with immediate
- head trauma responsible for intracranial hemorrhage
- severe IUGR (PN < 1800g)
- child whose death is foreseeable in the short term
- parental refusal
- child born under X
- absence de recueil du sang de cordon.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 2
- Occurrence of clinical or paraclinical adverse events during the child's follow-up (short- and long-term), attributable to the injected cell preparation
- Feasibility of the study: percentage of children included for whom the cell therapy procedure could be completed according to the required quality criteria.
Secondary endpoints 1
- - Preliminary efficacy as measured by neurodevelopmental function till 2 years
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
Autologous cord blood-derived viable nuclear cells 50E6/kg prep
PRD11419314 · Product
- Active substance
- Autologous Cord Blood-Derived Mononuclear Cells
- Substance synonyms
- Autologous mononuclear cells derived from human cord blood, Hau-UCB-mnc
- Pharmaceutical form
- SUSPENSION FOR IV INFUSION
- Route of administration
- INTRAVENOUS USE
- Max daily dose
- 500000000 CFU/g colony forming unit(s)/gram
- Max total dose
- 100000000 CFU/g colony forming unit(s)/gram
- Max treatment duration
- 3 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- APHM
- Paediatric formulation
- No
- Orphan designation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Centre Hospitalier Regional De Marseille
- Sponsor organisation
- Centre Hospitalier Regional De Marseille
- Address
- 147 Boulevard Baille
- City
- Marseille
- Postcode
- 13005
- Country
- France
Scientific contact point
- Organisation
- Centre Hospitalier Regional De Marseille
- Contact name
- INVESTIGATOR
Public contact point
- Organisation
- Centre Hospitalier Regional De Marseille
- Contact name
- project manager
Locations
1 EU/EEA country · 12 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| France | Ongoing, recruiting | 20 | 12 |
| Rest of world | — | 0 | — |
Investigational sites
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| France | 2020-02-05 | 2020-02-05 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 5 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | 2017005927-12 PROTOCOLE V6_20240118_NEOSTEM | 6 |
| Recruitment arrangements (for publication) | 2017-005927-12 _Recruitment arrangements_NEOSTEM | 1 |
| Subject information and informed consent form (for publication) | 2017-002927-12 NIFC_20190205_NEOSTEM | 3 |
| Subject information and informed consent form (for publication) | 2017-002927-12 NIFC_mise en banque20190205_NEOSTEM | 1 |
| Synopsis of the protocol (for publication) | 2017002927-12 RESUME_20190209_NEOSTEM | 1 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-08-07 | France | Acceptable 2024-09-03
|
2024-09-10 |